Randomized Pilot Study for the Treatment of Cutaneous Leiomyomas With Botulinum Toxin
- Cutaneous leiomyomas are smooth muscle tumors that may arise sporadically or in
association with an inherited cancer-related genodermatosis.
- Leiomyomas may be severely painful, and current management is generally unsatisfactory.
- Studies demonstrate increased nerve density within and around leiomyomas as well as
increased acetylcholinesterase staining of associated nerves.
- Botulinum toxin-A has been used in the treatment of pain syndromes.
- Based on the known mechanisms of action of botulinum toxin-A, treatment with botulinum
toxin-A (BOTOX; Allergan, Inc.), may ameliorate the severe paroxysmal pain of
symptomatic cutaneous leiomyomas.
- Primary: To assess change in worst lesional pain in the past week based on Brief Pain
Inventory (BPI) from Week 0 to Week 4 in treated patients versus controls.
- Primary: To assess improvement in pain based on Visual Analog Scale (VAS) after
application of ice at Week 4 compared to baseline in treated patients versus controls.
- Secondary: To assess change in magnitude and in frequency of painful episodes based on
a weekly patient diary in treated patients versus controls.
- Secondary: To assess persistence of pain control at Weeks 12 based on the BPI and VAS.
- Secondary: To assess the frequency of rescue pain medication use in treated patients
versus controls during the 24 week study period.
- Secondary: To determine the impact of leiomyoma treatment on quality of life.
- Secondary: To assess change in patient's condition based on the Patient Global
Impression of Change.
- Secondary: To evaluate the immunohistochemical staining of nerve fibers and muscle in
cutaneous leiomyomas in control and treated lesions following the conclusion of the
- Subjects greater than or equal to18 years with at least 1 symptomatic cutaneous
- Pain symptoms must occur at least once a week and be characterized as greater than or
equal to 5 out of 10.
- A 12-week double-blind placebo controlled pilot study of 18 subjects with symptomatic
leiomyomas will include initial assessment with BPI, photography, and skin biopsies,
followed by treatment of subjects who initially received placebo.
- Cutaneous leiomyomas will undergo intralesional injection with botulinum toxin-A.
- Subjects will return at Weeks 4 and 12 for repeat assessment using pain and quality of
life questionnaires and photography. Skin biopsies will be performed at week 12.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double-Blind, Primary Purpose: Treatment
Worst lesional pain as measured by the Brief Pain Inventory (BPI) at baseline and at 4 and 12.
Edward W Cowen, M.D.
National Cancer Institute (NCI)
United States: Federal Government
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Bethesda, Maryland 20892|